Inovio Gets FDA Approval for Rolling BLA Submission for Rare Disease Asset INO-3107

Inovio Pharmaceuticals has received FDA approval to submit a rolling BLA for its rare disease asset, INO-3107, for Recurrent Respiratory Papillomatosis. The company projects completion of the submission in the next few months.

INOVIO Pharmaceuticals, Inc. (NASDAQ: INO) has announced that it has received FDA approval to begin a rolling submission of its Biologics License Application (BLA) for INO-3107, an investigational DNA medicine designed to treat Recurrent Respiratory Papillomatosis (RRP) in adults. The company aims to complete the submission in the coming months and requests priority review from the FDA, with the goal of file acceptance by the end of 2025 [1].

INOVIO's INO-3107 is an antigen-specific T cell response activator against HPV-6 and HPV-11 proteins. A Phase 1/2 trial, RRP-001, demonstrated significant efficacy, with 72% of patients showing a 50-to-100% reduction in the number of surgeries after one year of treatment. A subsequent retrospective study, RRP-002, indicated that this number increased to 86% after two years, with half of the participants requiring no additional surgeries [1].

The FDA has granted INO-3107 both Orphan Drug and Breakthrough Therapy designations, enabling the company to submit its BLA under the FDA's accelerated approval program. INOVIO has also received CE marking for its CELLECTRA® delivery device in the EU, facilitating commercialization in the region [1].

INOVIO's DNA medicines platform leverages precisely designed DNA plasmids delivered by its proprietary CELLECTRA device. This platform is designed to optimize the design and delivery of innovative DNA medicines that teach the body to manufacture disease-fighting tools. The company's technology is focused on treating and protecting people from HPV-related diseases, cancer, and infectious diseases [1].

The FDA's recent activity in recognizing the importance of accelerating the approval of new technologies for rare diseases like RRP is encouraging for INOVIO. The company's goal is to bring INO-3107 to market as quickly as possible, potentially becoming the preferred treatment for RRP patients and providers [1].

References:
[1] https://ir.inovio.com/news-releases/news-releases-details/2025/INOVIO-to-Begin-Rolling-Submission-of-BLA-for-INO-3107-for-the-Treatment-of-RRP-in-Adults/default.aspx